C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/62 (2006.01) A61K 31/203 (2006.01) A61K 31/7088 (2006.01) A61K 38/06 (2006.01) A61K 38/17 (2006.01) A61K 38/18 (2006.01) A61K 39/395 (2006.01) A61K 47/48 (2006.01) A61K 48/00 (2006.01) C07K 19/00 (2006.01) C12N 15/63 (2006.01) C12P 21/00 (2006.01)
Patent
CA 2445577
Targeted therapeutics that localize to a specific subcellular compartment such as the lysosome are provided. The targeted therapeutics include a therapeutic agent and a targeting moiety that binds a receptor on an exterior surface of the cell, permitting proper subcellular localization of the targeted therapeutic upon internalization of the receptor. Nucleic acids, cells, and methods relating to the practice of the invention are also provided.
La présente invention se rapporte à des traitements ciblés qui sont localisés au niveau d'un compartiment intracellulaire spécifique, tel que le lysosome. Ces traitements ciblés comprennent un agent thérapeutique ainsi qu'un fragment de ciblage qui se lie à un récepteur sur une surface extérieure de la cellule, ce qui permet une localisation intracellulaire correcte du traitement ciblé dès l'internalisation du récepteur. Cette invention concerne également des acides nucléiques, des cellules ainsi que des méthodes relatives à la mise en oeuvre de la présente invention.
Beverley Stephen M.
Lebowitz Jonathan H.
Biomarin Pharmaceutical Inc.
Fasken Martineau Dumoulin Llp
Symbiontics Inc.
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